The first patient has been dosed in a first-in-human clinical trial of a treatment for geographic atrophy (GA) secondary to age-related macular degeneration (AMD). 

CTx001, developed by biotechnology company Complement Therapeutics, is being evaluated in the Phase I/II Opti-GAIN study evaluating its safety, tolerability and preliminary efficacy. 

The treatment is an investigational AAV2-based gene therapy designed to transduce retinal cells with a construct encoding mini-CR1, a truncated and secreted form of Complement Receptor 1 that can modulate both the alternative and classical complement pathways.  

 Subretinal delivery of CTx001 enables local retinal production of mini-CR1, while the small size of mini-CR1 may support penetration across Bruch’s membrane resulting in broad ocular biodistribution, including the choriocapillaris. 

The Opti-GAIN study is being advanced alongside Pre-GAIN, Complement Therapeutics’ ongoing natural history study in GA currently enrolling in both the US and the UK. The studies are intended to support patient selection and the evaluation of novel structural and functional endpoints, including ellipsoid zone (EZ) and focal Optical Coherence Tomography (OCT)-based microperimetry. 

“Dosing the first patient in Opti-GAIN is an important milestone for Complement Therapeutics and for the advancement of CTx001 in Geographic Atrophy,” said Dr Rafiq Hasan, Chief Executive Officer of Complement Therapeutics.  

“We believe the combination of a differentiated asset and an integrated clinical development strategy sets CTx001 apart. By advancing Opti-GAIN alongside Pre-GAIN, we are generating natural history and interventional data to better inform patient selection, endpoint strategy and future clinical development.” 

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